Acute Myeloid Leukemia (AML)NCT01962636

Umbilical Cord Blood Transplantation Using a Myeloablative Preparative Regimen for Hematological Diseases

A study testing Fludarabine, Cyclophosphamide and Total Body Irradiation in people with Acute Myeloid Leukemia (AML) and Acute Lymphocytic Leukemia (ALL), run by Masonic Cancer Center, University of Minnesota at 1 site in United States.

Terminated

Stopped early. The study ended before it was planned to and will not resume.

Official record

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Trial summary

Who is it for?: Ages Up to 55 years
Conditions the study is for: Acute Myeloid Leukemia (AML), Acute Lymphocytic Leukemia (ALL), Chronic Myelogenous Leukemia
What is being tested?: Fludarabine, Cyclophosphamide and Total Body Irradiation
Type of treatment: Injection or infusion, Surgery or procedure
Could I receive a placebo?: No, everyone receives an active treatment
How many people: 200 participants
Where: 1 siteUnited States
Study dates: Dec 2016to 14 May 2026
Phase: Interventional

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

Ages Up to 55 years
All sexes
No healthy volunteers
Child, Adult

Who may be able to join

Eligible Disease Status
Acute Myeloid Leukemia (AML): high risk CR1 (as evidenced by preceding MDS, high risk cytogenetics, ≥ 2 cycles to obtain CR, erythroblastic or megakaryocytic leukemia; CR2+. All patients must be in CR as defined by hematological recovery, AND <5% blasts by light microscopy within the bone marrow with a cellularity of ≥15%.
Very high risk pediatric patients with AML: Patients <21 years, however, are eligible with (M2 marrow) with < 25% blasts in marrow after having failed one or more cycles of chemotherapy.
Acute Lymphocytic Leukemia (ALL): high risk CR1 as defined by cytogenetics (such as t(9;22), t (1:19), t(4;11), other MLL rearrangements, hypodiploidy, or IKZF1 abnormalities), DNA index < 0.81, > 1 cycle to obtain CR or presence minimal residual disease (MRD). Patients in CR2+ are eligible. All patients must be in CR as defined by hematological recovery, AND <5% blasts by light microscopy within the bone marrow with a cellularity of ≥15%.
Very high risk pediatric patients with ALL: patients <21 years are also considered high risk CR1 if they had M2 or M3 marrow at day 42 from the initiation of induction or M3 marrow at the end of induction. They are eligible once they achieved a complete remission.
Chronic Myelogenous Leukemia excluding refractory blast crisis: To be eligible in first chronic phase (CP1) patient must have failed or be intolerant to imatinib mesylate.

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Plasma Cell Leukemia after initial therapy, who achieved at least a partial remission
Advanced Myelofibrosis
Myelodysplasia (MDS) IPSS INT-2 or High Risk (i.e. RAEB, RAEBt) or Refractory Anemia with severe pancytopenia or high risk cytogenetics: Blasts must be < 10% by a representative bone marrow aspirate morphology.
Chronic Lymphocytic Leukemia/Small Lymphocytic Lymphoma (CLL/SLL), Marginal Zone B-Cell Lymphoma or Follicular Lymphoma are eligible if there was disease progression/relapse within 12 of achieving a partial or complete remission. Patients who had remissions lasting > 12 months, are eligible after at least two prior therapies. Patients with bulky disease (nodal mass greater than 5 cm) should be considered for de-bulking chemotherapy before transplant.
Lymphoplasmacytic Lymphoma, Mantle-Cell Lymphoma, Prolymphocytic Leukemia are eligible after initial therapy in CR1+ or PR1+.
Large Cell NHL > CR2/> PR2: Patients in CR2/PR2 with initial short remission (<6 months) are eligible.
Lymphoblastic Lymphoma, Burkitt's Lymphoma, and other high-grade NHL after initial therapy if stage III/IV in CR1/PR1 or after progression if stage I/II < 1 year.
Multiple Myeloma beyond PR2: Patients with chromosome 13 abnormalities, first response lasting less than 6 months, or β-2 microglobulin > 3 mg/L, may be considered for this protocol after initial therapy.
Myeloproliferative Syndromes
Availability of suitable UCB unit(s)
0 to 55 years
Voluntary written consent (adult or parental/guardian)

Who cannot join

previous irradiation that precludes the safe administration of TBI - Radiation Oncology will evaluate all patients who have had previous radiation therapy
chemotherapy refractory large cell and high grade NHL (ie progressive disease after > 2 salvage regimens)
if ≤ 18 years old, prior myeloablative transplant within the last 6 months. If >18 years old prior myeloablative allotransplant or autologous transplant
extensive prior therapy including > 12 months alkylator therapy or > 6 months alkylator therapy with extensive radiation
pregnant or breastfeeding
HIV positive

Trial locations and contacts

1 site in 1 country.

United States1 site

University of Minnesota Masonic Cancer Center
Minneapolis, Minnesota, 55455

Other trials in United States

About this study

Official title: Umbilical Cord Blood Transplantation Using a Myeloablative Preparative Regimen for the Treatment of Hematological Diseases

This is a treatment guideline for an unrelated umbilical cord blood transplant (UCBT) using a myeloablative preparative regimen for the treatment of hematological diseases, including, but not limited to acute leukemias. The myeloablative preparative regimen will consist of cyclophosphamide (CY), fludarabine (FLU) and fractionated total body irradiation (TBI).

Read the detailed description

This is a study to collect routine clinical data from UCBT using unrelated single or double UCB units as an alternative, non-HLA-matched stem cell source for patients with hematological diseases.

data collection from transplant preparative therapy consisting of treatments with chemotherapeutic regimens and total body irradiation.
data collection from umbilical cord blood selection and infusion.
data collection from standard supportive disease and transplant related care.

Pre- and post-transplant medication, UCB selection and infusion, supportive care, and follow-up will be according to the current University of Minnesota BMT guidelines.

An average of 18 patients are expected to be treated on this protocol per year.

Conditions: Acute Myeloid Leukemia (AML), Acute Lymphocytic Leukemia (ALL), Chronic Myelogenous Leukemia, Plasma Cell Leukemia, Myelofibrosis, Myelodysplasia
Being tested: Fludarabine, Cyclophosphamide, Total Body Irradiation, Cyclosporine A, Mycophenylate mofetil
Keywords: Umbilical Cord Transplant, Acute Myeloid Leukemia (AML), Acute Lymphocytic Leukemia (ALL), Chronic Myelogenous Leukemia, Plasma Cell Leukemia, Myelofibrosis, Myelodysplasia, Chronic Lymphocytic Leukemia, Small Lymphocytic Lymphoma, Marginal Zone B-Cell Lymphoma, Follicular Lymphoma, Lymphoplasmacytic Lymphoma, Mantle-Cell Lymphoma, Prolymphocytic Leukemia, Large Cell Non-Hodgkin Lymphoma, Lymphoblastic Lymphoma, Burkitt's Lymphoma, Non-Hodgkin Lymphoma, Multiple Myeloma
Medical subject headings: Leukemia, Myeloid, Acute, Precursor Cell Lymphoblastic Leukemia-Lymphoma, Leukemia, Myelogenous, Chronic, BCR-ABL Positive, Leukemia, Plasma Cell, Primary Myelofibrosis, Anemia, Refractory, with Excess of Blasts, Leukemia, Lymphocytic, Chronic, B-Cell, Lymphoma, B-Cell, Marginal Zone

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

FludarabineDrug
Also called Fludara
25 mg/m^2 IV of Fludarabine will be given over 1 hour on days -8, -7, and -6 pre-UCB transplant.
CyclophosphamideDrug
Also called Cytoxan
60 mg/kg IV of Cyclophosphamide will be given over 2 hours on days -7 and -6 pre-UCB transplant.
Total Body IrradiationRadiation
165 cGy of total body irradiation will be given twice a day on days -4, -3, -2, and -1.
Cyclosporine ADrug
Also called CSA
Cyclosporine A (CSA) will start day -3 and will be administered PO/IV maintaining a trough level between 200 and 400 ng/mL. For adults the initial dose will be 2.5 mg/kg IV over 1 hour every 12 hours. For children < 40 kg the initial dose will be 2.5 mg/kg IV over 1 hour every 8 hours.
Mycophenylate mofetilDrug
Also called MMF
Mycophenylate mofetil (MMF) 3 gram/day IV/PO for patients who are ≥ 40 kg divided in 2 or 3 doses. Pediatric patient (<40 kilograms) will receive MMF at the dose of 15 mg/kg/dose every 8 hours beginning day -3.
Umbilical cord bloodBiological
Also called UCB
Pre-medications and UCB infusion will be per current institutional policies/guidelines. The infusion of the first UCB unit should begin within 15 minutes, and no later than 30 minutes after arrival on the Unit. If 2 units are used, both cords will be infused within 30-60 minutes of each other as deemed clinically safe by the BMT attending or designee.

Study groups in NCT01962636
Study group	Role	What they receive
Umbilical Cord Blood Transplant	Experimental	The myeloablative preparative regimen will consist of cyclophosphamide (CY), fludarabine (FLU) and fractionated total body irradiation (TBI)followed by umbilical cord blood transplant. Immunosuppressive Cyclosporine and Mycophenylate Mofetil (MMF) will be administered pre- and post UCBT. Receives: Drug: Fludarabine; Drug: Cyclophosphamide; Radiation: Total Body Irradiation; Drug: Cyclosporine A; Drug: Mycophenylate mofetil; Biological: Umbilical cord blood

What is being measured

The primary outcome is the main question the study is designed to answer.

Survival at 1 year post-transplant
The number of patients that are still living 1 year after UCBT.
Measured: 1 year

Secondary outcomes (9)

Incidence of neutrophil engraftment at day 42.
Number of subjects with neutrophil engraftment at day 42 post UCBT.
Measured: 42 days
Platelet engraftment at 1 year.
Number of patients with platelet engraftment at 1 year post UCBT.
Measured: 1 year
Pattern of chimerism after transplant.
Pattern of chimerism after transplant. Chimerism will be plotted with box-plots and described over time.
Measured: 1 year
Incidence of graft failure.
Cumulative incidence of graft failure after UCBT.
Measured: 100 days
Incidence of acute graft versus host disease at 100 days.
Cumulative incidence will be used to estimate acute graft versus host disease 100 days after UCBT.
Measured: 100 days
Incidence of chronic graft versus host disease at 1 year.
Cumulative incidence will be used to estimate chronic GVHD at 1 year post UCBT.
Measured: 1 year
Incidence of transplant related mortality at 6 months.
Cumulative incidence will be used to estimate transplant related mortality at 6 months post UCBT.
Measured: 6 months
Incidence of disease free survival
Kaplan-Meier curves will be used to estimate disease-free survival at 1 and 2 years post UCBT.
Measured: 1, 2 years
Incidence of overall survival.
Kaplan-Meier curves will be used to estimate overall survival at 1 and 2 years post UCBT.
Measured: 1, 2 years

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

Dec 201614 May 2026

First posted14 Oct 2013 (estimated)
Study startDec 2016
Primary completion14 May 2026
Study completion14 May 2026

Why it stopped: No longer a research question to answer.

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Other trials for Acute Myeloid Leukemia (AML)Studies of the same condition, open and under way.
What the trial phases meanHow a treatment moves from first tests in people to approval.

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design

Study type: Interventional
Phase: Phase N/A
Primary purpose: Treatment
Allocation: Not applicable
Intervention model: Single group
Masking: None (open label)
Enrolment: 200 participants (estimated)

IDs, dates and publications

ClinicalTrials.gov ID: NCT01962636
Sponsor's protocol ID: 2013OC013
Status last verified: May 2026
First submitted: 10 Oct 2013
Last updated: 20 May 2026
Results posted: No
Data monitoring committee: No

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

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